Ignite Creation Date:
2024-05-06 @ 3:06 PM
Last Modification Date:
2024-10-26 @ 1:43 PM
Study NCT ID:
NCT04525352
Status:
TERMINATED
Last Update Posted:
2022-07-13
First Post:
2020-08-11
Brief Title:
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
Sponsor:
Rocket Pharmaceuticals Inc
Organization:
Rocket Pharmaceuticals Inc
Study Overview
Official Title:
A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis IMO to Evaluate the Safety and Preliminary Efficacy of Autologous CD34 Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene
Status:
TERMINATED
Status Verified Date:
2022-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Study discontinued due to feasibility
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product IP RP-L401
Detailed Description:
This is a non-randomized Phase 1 study to evaluate the preliminary safety and efficacy of hematopoietic gene therapy consisting of autologous CD34 enriched hematopoietic cells transduced with the lentiviral vector LV carrying the human TCIRG1 transgene RP-L401 in pediatric patients with IMO Following myeloablative conditioning patients will receive an infusion of the genetically modified hematopoietic stem and progenitor cells HSPCs
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None